Strongbridge Biopharma (SBBP)
SBBP is a global commercial-stage biopharmaceutical company focused on the development and commercialization of therapies for rare diseases with significant unmet needs. Their first commercial product is Keveyis (dichlorphenamide), the first and only treatment approved by the FDA for hyperkalemic, hypokalemic, and related variants of primary periodic paralysis (PPP), a group of rare hereditary disorders that cause episodes of muscle weakness or paralysis. Their second commercial product, Macrilen (macimorelin) is an oral growth hormone secretagogue receptor agonist, and is the first and only oral drug approved by the FDA for the diagnosis of patients with adult growth hormone deficiency (AGHD). They recently (10/31/18) entered into an agreement for NVO to acquire US and Canadian rights to Macrilen. Additionally, they have two clinical-stage product candidates for rare endocrine diseases, Recorlev and Veldoreotide. Recorlev (levoketoconazole) is a cortisol synthesis inhibitor currently being studied for the treatment of endogenous Cushing’s syndrome. Veldoreotide is a next-generation somatostatin analog being investigated for the treatment of acromegaly and potential additional applications in other conditions amenable to somatostatin receptor activation. Both Recorlev and veldoreotide have received orphan designation from the FDA and the EMA.